An observational study of 329,000 Medicare admissions found that older persons receiving hospital care from allopathic (M.D.) or osteopathic (D.O.) physicians experience similar quality and cost of care. Researchers also highlight systemic health inequities faced by persons with sickle cell disease.
Researchers successfully demonstrate AAV vector efficacy in aged animal models, showing robust hearing rescue in mice with a mutation equivalent to a defective human gene. The study suggests that virally mediated gene therapy could potentially treat genetic hearing loss, especially for patients diagnosed at advanced age.
Researchers have discovered that HER3 plays a crucial role in promoting cell survival in metastatic colorectal and pancreatic cancer. The surrounding liver microenvironment activates HER3, making it an emerging therapeutic target for these types of cancer.
A new study demonstrates that subretinal delivery of AAV-RPE65 gene supplementation increases RPE65 isomerase activity and recovers chromophore 11-cis retinal after bleaching in mice with D477G RPE65 mutation, providing initial proof-of-principle data for gene supplementation as a treatment.
Gene therapy is being tested for rare skeletal dysplasia, affecting 1 in 200,000. Patients will receive a one-time infusion of viral vector containing correct gene sequence.
Increased expression of Musashi 1 on breast cancer cells has significant implications for understanding dormancy and survival in bone marrow. Msi 1 knockdown led to a reduction in cancer stem cells with undetectable PD-L1, suggesting a potential therapeutic target.
Researchers used cancer proteomics data to identify gene candidates for therapeutic targeting, focusing on protein kinases in uterine endometrial cancer cells. Public molecular resources and multi-omics data analysis can prioritize genes of interest for future studies.
Researchers developed a size exclusion chromatography (SEC) method to measure empty capsids in gene therapy programs, outperforming other analytical methods. The study found SEC-MALS to be linear, accurate, and precise, providing a robust analytical technique for advancement of gene therapy programs.
The LY6 gene family has been found to be overexpressed in uterine corpus endometrial carcinoma (UCEC), leading to poor patient survival. Several LY6 genes have been identified as potential tumor-associated antigens and biomarkers for UCEC detection and prognosis.
Scientists have developed a new method to deliver genetic information to stem cells using nanoparticles coated with a specific polymer, enabling more efficient control over cellular differentiation. This innovation has the potential to improve the efficiency and effectiveness of regenerative medicine treatments.
Researchers identified three novel dual-purpose therapeutic targets using PandaOmics, which could treat both aging and glioblastoma multiforme. The target hypotheses include cyclic nucleotide gated channel subunit alpha 3 (CNGA3), glutamate dehydrogenase 1 (GLUD1) and sirtuin 1 (SIRT1).
Researchers at Brigham and Women's Hospital have identified a potential therapeutic target and developed a unique delivery system to treat osteosarcoma. MicroRNA nanoparticles delivered locally using a hydrogel suppressed osteosarcoma growth while decreasing bone damage.
Researchers evaluated six AAV vectors in 12 preclinical liver models to identify the most relevant combination for safe and efficient gene therapy. The study proposes using multiple models to provide a more complete picture of vector function, informing clinical decisions.
Researchers successfully delivered stabilized divalent siRNA molecules to animal models that blocked SARS-CoV-2 and prevented infection. The technology is adaptable for other pulmonary diseases such as pulmonary fibrosis and respiratory viruses.
A new therapy delivery method, using modified viruses engineered with fusogens Myomaker and Myomerger, shows promise as a treatment for Duchenne muscular dystrophy. The vector can deliver a vital gene needed for muscle function to cells, potentially providing a lifelong supply of the missing gene.
Scientists design a mini gene therapy to replace the mutated gene in Usher 1F, increasing protein production and restoring hearing in mice. The approach may also prevent blindness by targeting a different form of the protein involved in vision loss.
Researchers have developed a bioengineered AAV3B capsid variant with improved transduction to human liver cells and reduced seroreactivity to human serum samples. The variant, AAV3B-V04, demonstrates enhanced hepatocyte tropism and immune evasion, making it a promising candidate for gene therapy.
A new genetic therapy, BIIB080, has been shown to safely lower levels of the harmful tau protein in patients with Alzheimer's disease. The trial found a greater than 50% reduction in tau protein concentrations after 24 weeks in treatment groups.
Cedars-Sinai investigators have discovered a novel way to treat amyotrophic lateral sclerosis (ALS) and retinitis pigmentosa using human induced pluripotent stem cells. The new approach uses cells derived from iPSCs that are renewable, scalable, and can delay disease progression in rodents.
A novel gene therapy approach decreases intraocular pressure in pre-clinical models of glaucoma. The treatment uses a viral vector to deliver instructions to cells, producing an enzyme that helps reduce fluid accumulation and pressure. This breakthrough holds promise for patients with glaucoma.
Researchers have created a drug-like cocktail that successfully regenerated hair cells in a mouse model by reprogramming genetic pathways within the inner ear. The approach could lead to clinical trials for a gene therapy that can be administered to people with hearing loss, potentially treating up to 90% of cases.
Researchers have built a new model to examine Usher Syndrome, a leading cause of combined deafness and blindness. The model replicates the visual problems not addressed by previous models, offering insight into strategies for designing therapeutic interventions.
Researchers at Tokyo Medical and Dental University have found that a specific lipid, alpha-tocopherol, increases the uptake of antisense oligonucleotides in the stroke-lesioned brain. This delivery method has potential for targeted protein expression after a stroke.
Researchers at the University of Tokyo have discovered the 3D structure of TnpB, a protein involved in genome editing and a probable precursor to the CRISPR-Cas12 enzyme. The study reveals how TnpB recognizes and cuts DNA using a unique pseudoknot shape similar to that found in guide RNAs of Cas12 enzymes.
Researchers at Indiana University School of Medicine investigated the protein BVES and its crucial role in muscular dystrophy. They discovered that inhibiting proteasome degradation with Bortezomib can alleviate symptoms of BVES-deficient muscular dystrophy.
Researchers at Children's Hospital of Philadelphia discovered that viral proteins use phase separation to coordinate the complex process of replicating viral genomes and then encapsulating them in a viral particle. This process allows for the orderly and coordinated formation of infectious viral offspring.
A recent study found that a tiny fragment of the PC1 protein can suppress the disease in mouse models, reducing cyst size and revealing new avenues for developing therapies. The researchers discovered that this small piece interacts with a mitochondrial protein called NNT, opening up new avenues to study its normal function.
Researchers developed a programmable bacterial injection system that can deliver a range of proteins, including those for gene editing, to different cell types. The system has shown promising results in targeting cancer cells and delivering proteins to the brain in live mice without provoking an immune response.
The NCATS Platform Vector Gene Therapy (PaVe-GT) program utilizes FDA guidance to prepare applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), stimulating gene therapy development. The PaVe-GT program has already helped receive ODD and RPDD designations for a gene therapy product treating PCCA-r...
Researchers have found that valosin-containing protein (VCP) is essential for KRAS-mutant pancreatic ductal adenocarcinoma cell growth and survival. Inhibiting VCP, combined with autophagy inhibition, enhances efficacy in preclinical studies.
Researchers at Tokyo Medical and Dental University have developed a polymeric nanoparticle gene delivery system that promotes bone formation after traumatic inflammation. The therapy inhibits excessive inflammation and prevents residual ridge resorption, leading to improved tissue healing after tooth extraction.
Researchers at Texas A&M University have developed the first molecular therapeutic for Angelman syndrome, a devastating neurogenetic disorder. The therapy targets an evolutionarily conserved region in the UBE3A-AS transcript and has shown promising results in clinical trials.
Researchers used a monoclonal antibody to reduce circulating IgG levels, allowing for successful gene delivery to the liver and heart via systemic AAV-based gene therapy in mice and non-human primates. The strategy has potential to circumvent neutralizing antibody limitations and expand gene therapy accessibility.
Researchers at Harvard University developed a novel RNA sense-and-respond circuit, DART VADAR, which utilizes an enzyme to detect specific molecular markers of disease and cell types. This enables highly specific treatments for various diseases by triggering the translation of therapeutic genetic payloads.
Researchers at UCLA successfully used base editing to correct a mutation causing rare immune deficiency CD3 delta SCID. The treatment corrected an average of 71% of patient stem cells and allowed them to produce fully functional T cells, suggesting long-term persistence of corrected blood stem cells.
A new study published in Blood Advances found that the average life expectancy of publicly insured individuals with sickle cell disease is roughly 52.6 years, which is significantly lower than the overall US life expectancy of 73.5 years for men and 79.3 years for women. The study also revealed worse survival outcomes among those insur...
Researchers have designed a new RNA sensor that can selectively activate synthetic genes in specific cells, opening up possibilities for targeted therapies for cancer and other diseases. The system uses an enzyme that naturally exists in most animal cells to detect and repair mismatches in double-stranded RNA.
Researchers have successfully used AAV1.NT-3 gene therapy to improve muscle physiology and prevent age-related sarcopenia in mice. The treatment resulted in restored muscle mass, strength, and neural connections, offering a potential new option for managing this debilitating condition.
Pancreatic cancer is an incurable form of cancer, but gene therapies are being developed to treat it. The article reviews ongoing clinical trials and discusses various biotherapies, including vaccines and CAR-T cell therapy.
Gene therapy using CRISPR-Cas9 lipid nanoparticles has been shown to be highly effective in reducing target protein expression in mice. The new delivery system increases the efficiency of in vivo gene therapy, paving the way for safe and effective treatment.
Researchers found that severe herpesvirus infections can strongly activate host cellular immunity, leading to a therapeutic effect on refractory adult T-cell leukemia/lymphoma. This activation may play an important role in the survival of patients with this intractable disease.
Researchers have developed a new gene therapy to treat alpha 1-antitrypsin deficiency, an autosomal recessive disorder that causes emphysema. The therapy uses an adeno-associated virus (AAV) vector coding for an oxidation-resistant human α1-antitrypsin protein.
Researchers at UTHSC are working on a project to find the first therapeutic intervention to prevent frontotemporal dementia or slow its progression in a mouse model linked with the condition. They aim to use DNAzymes to target pathological tau aggregates, which cause cognitive impairment and progressive neuropathological symptoms.
A recent study published in Immunity reveals that human T-cell receptor genes exhibit unexpectedly high variability among individuals, with each person having a unique set of gene variants. The researchers identified 175 new gene variants originating from Neanderthals, which are present in up to 20% of modern humans in Europe and Asia.
Researchers from Rice University have developed a new approach to control gene expression using proteolysis targeting chimeras (PROTACs). By reengineering the PROTAC molecular infrastructure, they demonstrated the ability to achieve chemically induced dimerization (CID), allowing for precise control over gene activation in specific loc...
Researchers evaluate an integrated NGS system, delivering accurate diagnoses in under 24 hours and expanding targeted treatments available to patients with myeloid neoplasms. The assay identified 80-92% of genetic variants, demonstrating promising results for accelerating precision therapies.
Scientists have identified genes that play key roles in the development of coronary artery disease (CAD), a leading cause of death worldwide. The study found notable differences in gene activity between males and females, as well as between cells that were multiplying and those that were not.
Researchers from HKUMed discovered that somatic deletion of AKTIP promotes luminal breast cancer development and resistance to endocrine therapy. Blocking the alternative escape pathway with a JAK2/STAT3 inhibitor can overcome this resistance, offering new therapeutic possibilities for patients with AKTIP gene deletion.
Researchers discover gene therapy ophNdi1 that boosts mitochondrial performance in retinal ganglion cells, potentially treating glaucoma and age-related macular degeneration. The therapy shows protective effects in three models of mitochondrial dysfunction.
A novel form of chemoimmunotherapy has been proven to be a promising treatment for canine cancer, extending the lives of terminally ill dogs. The therapy uses modified Mesenchymal Stem Cells carrying a potent 'kill-switch' that induces anti-cancer immunity and improves quality of life.
Researchers have discovered an anti-ageing gene that can rewind the heart's biological age by 10 years, offering a potential target for patients with heart failure. The study found that administering the healthy mutant gene to elderly patients with severe heart problems improved their cardiac function and rejuvenated their immune system.
Researchers at Children's Hospital of Philadelphia have developed a custom-built application to automate the determination of engraftment after hematopoietic stem cell transplant. The tool has been shown to improve accuracy of reported engraftments, reducing errors in neutrophil and platelet engraftment reporting.
Researchers at Massachusetts General Hospital created a new class of technologies called CRISPR-associated transposases (CASTs) to overcome diverse disease-causing mutations. The optimized approach improves product purity and genome-wide specificity, offering a potential solution for inserting entire genes into the genome.
Researchers found four genetic populations of the finless porpoise in Chinese waters and three in the Gulf of Thailand, with unique matriline lineages. The study suggests ongoing gene flow among populations, requiring careful evaluation of inter-population connectivity for conservation action.
Researchers at University of Texas M. D. Anderson Cancer Center develop a novel mRNA delivery system using extracellular vesicles, which can initiate collagen production in cells and has potential for other mRNA therapies
A new approach to gene therapy for inherited blindness uses lipid nanoparticles to deliver mRNA inside the eye, targeting light-sensitive cells and creating proteins that edit vision-harming gene mutations. The technology has shown promising results in animal studies, including mice and nonhuman primates.
A new gene therapy treatment has shown promising results in a clinical trial for Artemis-SCID patients, improving their immune function and reducing treatment complications. The treatment involves adding a healthy copy of the Artemis gene to the patient's own cells, resulting in improved T-cell and B-cell immunity.
The use of 3D-patient tumor avatars (3D-PTAs) is crucial for guiding treatment decisions in precision oncology. These avatars, including patient-derived organoids, 3D bioprinting, and microscale models, can accurately depict a tumor with its microenvironment, enabling the testing and prediction of therapeutic drug efficacy.
Scientists at Duke University have made a breakthrough in controlling gene expression in response to injury, using a segment of fish DNA called TREE. The method successfully targeted gene activity to specific regions and time windows, showing promise for regenerating damaged tissues in mammals.
Researchers from Japan have developed an RNA interference method using antisense oligonucleotides to correct a genetic defect in Fukuyama Muscular Dystrophy. This approach has shown promise in treating patients with the disease, which is characterized by generalized muscle weakness and intellectual disability.